A prospective multicenter observational post authorization safety sub-registry to characterize the long-term safety profile of commercial use of eliglustat (Cerdelga®) in adult patients with Gaucher disease (ELISAFE)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Study drug and medical condition

• Medicinal product name:
  CERDELGA
• Study drug International non-proprietary name (INN) or common name:
  ELIGLUSTAT
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AX10) eliglustat

  eliglustat
• Medical condition to be studied:
  Gaucher's disease

Population studied

• Short description of the study population:
  The target study population will include adult patients who are treated with, or plan to be treated with Cerdelga or Cerezyme as part of routine management of their GD.
• Age groups:
  • Adults (18 to < 46 years)
  • Adults (46 to < 65 years)
  • Adults (65 to < 75 years)
  • Adults (75 to < 85 years)
  • Adults (85 years and over)
• Estimated number of subjects:
  150

Study design details

• Study design:
  This is a safety sub-registry of the ICGG Gaucher Registry (DIRECC07009), a non-interventional, multicenter, prospective PASS of Cerdelga, a treatment approved in many countries for adult participants with GD1.
• Main study objective:
  1. To evaluate the long term safety of Cerdelga in real-world clinical practice; 2. To describe the utilization of Cerdelga (compliance/adherence of Health Care Providers):; - to the labelling with regard to CYP2D6 genotyping prior to initiation of Cerdelga; - to the Cerdelga label with regard to patients' CYP2D6 predicted phenotype; - to the labelling with regard to use in Gaucher disease type 1
• Comparators:
  Patients on Cerezyme will provide a control group to descriptively compare the safety profile of Cerdelga to that of Cerezyme.
• Outcomes:
  • Incidence rate of any AE. ; • Frequencies and percentages of timing of CYP2D6 genotyping (prior to or following) initiation of Cerdelga. ; • Frequencies and percentages of CYP2D6 predicted phenotype among Cerdelga-treated patients. ; • Frequencies and percentages of types of GD among Cerdelga-treated patients. ; • Incidence rate of any serious adverse event.
• Data analysis plan:
  Analysis will be performed on the Safety Set defined as all patients enrolled in the sub-registry who have received at least one dose of Cerdelga or Cerezyme following enrollment. The main analysis of interest will be the incidence of any AE report for Cerdelga treated patients during their participation in the subregistry, considering treatment status at sub-registry enrollment if applicable. Similar calculations will be performed for the Cerezyme treated patients. Summaries will also be presented for three categories of treatment status within each of the Cerdelga and Cerezyme groups. Data will be analyzed as available, missing data will not be imputed. All summaries will be descriptive, no formal hypothesis testing will be performed.
• Summary results:
  Overall, Cerdelga showed a safety profile consistent with that observed during the clinical trials in adults with GD1 and market experience.; ; The data shows that the compliance/adherence of HCPs to the Cerdelga label was very high with regard to use in GD1 adults, CYP2D6 genotyping prior to initiation of Cerdelga, and use in the appropriate CYP2D6 metabolizer groups.